Genetic therapy of diseases caused by the immune system,...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01) A61K 31/70 (2006.01) A61K 47/42 (2006.01) C12N 15/85 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2198462

The invention concerns a DNA sequence for the genetic therapy of diseases caused by the immune system. The essential components of the DNA sequence consist of an activator sequence, a promoter module and a gene for the active substance. The activator sequence is activated cell or virus-specifically and the promoter module regulates this activation specifically according to the cell cycle. The choice of activator sequence and active substance depends on the indications. The DNA sequence is introduced into a viral or non-viral vector, supplemented by a ligand having affinity for the target cell. Depending on the choice of activator sequence and active substance, the following conditions can be treated by administration of the DNA sequence: the defective formation of blood cells; autoimmune diseases and allergies as well as rejection reactions against transplanted organs; chronic arthritis; viral and parasitic infections as well as the prevention of viral, bacterial and parasitic infections; leukaemias.

L'invention concerne une séquence d'ADN pour la thérapie génétique de maladies dépendant du système immunitaire. La séquence d'ADN comprend, comme éléments essentiels, une séquence activatrice, un module promoteur et un gène pour la substance active. La séquence activatrice est activée spécifiquement, suivant la cellule ou le virus, le module promoteur réglant cette activation spécifiquement, en fonction du cycle cellulaire. Le choix de la séquence activatrice et de la substance active est en fonction du domaine d'indication. La séquence d'ADN est introduite dans un vecteur viral ou non viral, complété d'un ligand ayant une affinité pour la cellule cible. Suivant le choix de la séquence activatrice et de la substance active, les cas suivant peuvent être traités par administration de la séquence d'ADN: insuffisances dans la formation des cellules sanguines; maladies auto-immunitaires et allergies, ainsi que réactions de rejet à l'encontre d'organes transplantés; arthrites chroniques; infections virales et parasitaires, ainsi que prophylaxie des infections virales, bactériennes et parasitaires; leucémies.

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